Rare Daily Staff

Shire released positive results from a late-stage study of its experimental C1 esterase inhibitor therapy for Hereditary Angioedema, a rare genetic disease characterized by recurrent and potentially fatal swelling of extremities, the gastrointestinal tract, and upper airways.

This study met its primary endpoint and all key secondary endpoints. It showed a statistically significant reduction in the number of attacks with a single dose administered every three to four days.

The results from the study evaluated the efficacy and safety of SHP616, a subcutaneously administered C1 esterase inhibitor compared to placebo over two 14-week treatment periods in patients 12 years of age or older with symptomatic Hereditary Angioedema, or HAE.

HAE is a rare, genetic disorder estimated to affect about 1 in 10,000 to 1 in 50,000 people worldwide. It is caused by having insufficient amounts of C1-esterase inhibitor, a protein in blood plasma that plays a role in inflammation.

The condition results in recurrent, localized swelling. The areas of the body most commonly affected are the extremities, gastrointestinal tract, and upper airways. The swelling can be debilitating and painful, potentially impacting both work and education for people living with HAE. Swelling of the throat can be life-threatening due to asphyxiation.

SHP616 is an investigational treatment administered subcutaneously. Shire is investigating the treatment to prevent angioedema attacks.

In a commonly reported measure of effectiveness, SHP616 showed a median HAE attack rate reduction of 79 percent from Day 0 (entire treatment period) or 85 percent from Day 14 (after reaching steady state) compared to placebo. A total of 78 percent of patients experienced 50 percent or greater reduction in HAE attack rate (key secondary) compared to placebo. Some 38 percent of patients were attack free during their SHP616 treatment period, compared to 9 percent during the placebo period.

The 75 patients randomized in this study were required to have at least two HAE attacks per month in the three consecutive months prior to screening, and were representative of the full HAE disease spectrum.

There were no treatment-related serious adverse events or deaths reported in the study. The most common adverse events including viral upper respiratory tract infection, upper respiratory tract infection, and headache.

In June, CSL Behring won FDA approval for Haegarda, the first subcutaneous C1 Esterase Inhibitor to treat HAE.

September 11, 2017

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